Oncopeptides has launched their United States Expanded Access Program with melflufen (also called Melphalan Flufenamide) for relapsed or refractory patients who have had at least one proteasome inhibitor, one immunomodulatory drug and one anti-CD38 monoclonal antibody. These patients are considered triple-class refractory.
The Expanded Access Program was created after the company submitted a New Drug Application (NDA) to the FDA for accelerated approval of melflufen in combination with dexamethasone for the treatment of adult patients with triple-class refractory multiple myeloma.
The approval for the Expanded Access Program is supported by data from the Phase II HORIZON study which showed that melflufen in combination with dexamethasone, has a potential to provide benefit for hard-to-treat relapsed or refractory multiple myeloma, including patients with triple-class refractory multiple myeloma and patients with extramedullary disease.
The EAP program means that myeloma patients can use melflufen with dexamethasone as "compassionate use" while the drug application is under FDA review. Forty to fifty medical sites in the U.S. are expected to enroll 100-200 patients in the sEAPort program.
Melflufen (melphalan flufenamide) is a first-in-class peptide-drug conjugate (PDC) that targets specific cells (aminopeptidases) and rapidly releases alkylating agents into tumor cells. Melflufen is rapidly taken up by myeloma cells and quickly releases the toxic payload. Aminopeptidases are overexpressed in myeloma cells and are found at higher levels in more advanced myeloma and in myeloma with a higher mutational burden.
In the lab, melflufen is 50-fold more potent in myeloma cells than the chemo payload itself and shows activity against myeloma that is resistant to other myeloma treatments.
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